January 1998

Washington Report
Dr. Charles J. Coté
No abstract available

As you have seen from past articles on this Web site (read Dr. Coté's article on The ASA Update for Pediatrics, AnesthesiaWeb November 1997), the vast majority of drugs do not have pediatric labels, i.e., there is language such as "safety and efficacy not determined for pediatric patients less than 18 years of age". This has left the practitioner in the position of prescribing drugs based on the experience of a few investigators, often without the full benefit of pharmacokinetic and pharmacodynamic data and certainly without the stamp of approval from the FDA.

At present the FDA cannot force industry to perform pediatric studies, they can only "suggest", so the easy road for them is to say that they do not intend for the drug to be used in children. This lack of interest by industry in the care of children has also left us with the need to reformulate medications so that they are palatable for children and it has often forced us to use drugs with a minimum of useful information from small and often poorly controlled studies. Without good manufacturing standards, such "homemade" formulations could theoretically alter drug bioavailability.

Within the past several weeks President Clinton signed new Food and Drug Administration legislation S.830, now Public Law 105-115. This new legislation will require drug companies to perform at least one pediatric study for new drug applications and to perform at least one study of currently approved medications which are still on patent. The type of study will be determined through negotiation with the Secretary of Health and Human Services after a written request to the manufacturer. In exchange the government would grant an additional 6 months of patent exclusivity. Money speaks! This could represent a major victory for those of us who take care of children however there is still much work to do.

On the surface the legislation sounds good, however the pharmaceutical industry, through statements made by the president of the Pharmaceutical Research and Manufacturers of America (a lobby for drug companies), has not embraced this concept and even is so bold as to state that children will be injured in the preclinical trials and that this is a reason not to perform such studies. I guess I interpret this as an attempt to scare off the FDA into thinking that the industry has the pediatric patient's best interest in mind and that they are trying to protect children! How off base an argument could they have chosen since as a whole (with some noted exceptions) the industry has denied that children even exist as having need for their medications and have hidden behind language such as "we do not intend for this drug to be used on children".

If this legislation is gutted during the coming weeks it would in fact deny children all the benefits of new pharmaceutical discoveries that adults currently enjoy, i.e. the status quo or worse would continue for many more years to come. This is our one window of opportunity which has been almost 30 years in the making since Shirkey first coined the term of children being "therapeutic orphans" (J Pediatr 72:119-120, 1968).

The task that follows the preliminary legislation is to insert language that gets done what needs to be done, i.e., the development of medications in formulations acceptable to children with well defined age appropriate pharmacokinetic and pharmacodynamic data as well as bioequivalency of the manufactured pediatric formulations. It will be interesting to try and combine all those goals into one study as the law is currently written.

Another important but difficult task will be to define what is meant by "one pediatric study". It is my hope that at last the FDA will have been empowered to "encourage" industry to perform the appropriate studies in the age groups that need it most, infants and toddlers. The intent of the new legislation will have failed if industry is allowed to perform one pediatric study only to have it turn out to be a study of older children who are pharmacologically very similar to adults.

If any of you are in a position to influence the language of this new legislation please do not hesitate to apply pressure. Children are a very precious commodity and we as a society have not provided them with the same safety and efficacy data for medications as for adults which in turn has lead to significant morbidity and even mortality.

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